Novartis has announced that the US Food and Drug Administration (FDA) has granted crizanlizumab (SEG101) Breakthrough Therapy designation for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD).
Breakthrough Therapy designation is awarded to therapies that treat a serious or life-threatening condition and demonstrate a substantial improvement over existing treatments; the designation for crizanlizumab was based on positive results of the Phase II SUSTAIN trial, in which the drug reduced the median annual rate of VOCs leading to healthcare visits by 45.3%.
SCD is a debilitating genetic blood disorder that affects the shape of red blood cells and can make blood cells and blood vessels stickier than usual, which can cause VOCs - unpredictable and extremely painful events that can lead to serious acute and chronic complications.
The study also demonstrated that crizanlizumab - a humanised anti-P-selectin monoclonal antibody - significantly increased the percentage of patients who did not experience any VOCs.
Dr Samit Hirawat, head, Novartis oncology global drug development, commented: ”Painful sickle cell crises matter because they can disrupt patients' lives, and often require hospital visits and medical attention".
He added, "We look forward to working closely with the FDA over the coming months toward making crizanlizumab, a therapy that has the potential to prevent sickle cell pain crises, available in the US as soon as possible.”